Bad News For Fabry Patients (and Amicus Investors)

James Radke, PhD

Amicus Therapeutics announced they will not be submitted a New Drug Application (NDA) to the US Food and Drug Administration (FDA) for their drug migalastat to treat patients with Fabry disease.
In a press release, Amicus noted that they had “received final FDA minutes from the September pre-NDA meeting and has conducted additional follow-up interactions with the Agency this week. In conjunction with the Agency, Amicus is further evaluating several U.S. pathways including potentially generating additional data on migalastat's effect on gastrointestinal symptoms in Fabry disease to support submission requesting full approval as well as a Subpart H strategy. In addition, the Agency has requested further integration of existing clinical data across studies which will require more time to complete.”
The company also stated that do not anticipate submitting a FDA by the end of this year. As to when and what they will eventually submit is also unknown.
John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics said,"Amicus remains committed to making migalastat available to Fabry patients with amenable mutations in the U.S. as rapidly as possible. We are appreciative of the FDA's ongoing collaboration in this program."

What is Fabry Disease

Fabry disease is an X-linked lysosomal storage disorder that leads to excessive deposition of globotriaosylceramide (GL-3) throughout the body. Skin, eye, kidney, heart, brain, and peripheral nervous system are highly vulnerable.
The condition is caused by deficiency of the enzyme alpha-galactosidase A (alpha-Gal A) which degrades GL-3. The accumulation of GL-3 is believed to cause a variety of symptoms, including pain, kidney failure, and increased risk of heart attack and stroke.
In the video below, Jack Johnson of the Fabry Support & Information Group (FSIG) discusses the symptoms of this rare disease.


Amicus Therapeutics Provides U.S. Regulatory Update for Migalastat Monotherapy [press release]. Cranbury, NJ: Amicus Therapeutics; October 2, 2015.
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